Marfan Syndrome Clinical Trial
Official title:
Clinical Trial On The Effects Of Moderate Physical Activity On Health And Well-Being In Adolescents And Young Adults With Marfan Syndrome
NCT number | NCT05809323 |
Other study ID # | H-53417 |
Secondary ID | |
Status | Recruiting |
Phase | N/A |
First received | |
Last updated | |
Start date | September 1, 2023 |
Est. completion date | July 1, 2027 |
Marfan syndrome (MFS) is a distinctive connective tissue disorder that affects multiple organ systems including the heart, bones, ligaments, and eyes, and is associated with significant risk of aortic dissection. Given limited evidence from in-vitro studies, and theoretical concerns, the majority of patients with MFS are restricted from certain physical activities. The lack of exercise and deconditioning have detrimental effects including increasing weakness, joint pain, decreased endurance, and depressive symptoms. Given the significant paucity of data currently existing on the effects of exercise in humans with MFS, and the recent, optimistic findings in rodent models, this pilot trial was established to assess the effects of moderated dynamic exercise in adolescents and young adults with MFS.
Status | Recruiting |
Enrollment | 50 |
Est. completion date | July 1, 2027 |
Est. primary completion date | July 1, 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 10 Years to 25 Years |
Eligibility | Inclusion Criteria: - Male and female patients diagnosed with Marfan syndrome (defined by Ghent criteria and either a pathogenic variant in FBN1 or ectopia lentis) - Age 10-25 years at enrollment Exclusion Criteria: 1. History of aortic surgery 2. History of spinal surgery with implanted materials that may negatively impact MRI safety or imaging quality. 3. Diagnosis of major congenital heart disease (ASD, VSD, bicuspid aortic valve, and mitral valve prolapse will not qualify as exclusion criteria) 4. Condition limiting the ability to perform moderate exercise. 5. Major concurrent diagnosis that may confound the interpretation of the effect of the proposed intervention on the proposed outcome measures. 6. Aortic dilation meeting threshold for prophylactic aortic surgical intervention (>/= 4.5 cm in diameter) |
Country | Name | City | State |
---|---|---|---|
United States | Texas Children's Hospital | Houston | Texas |
Lead Sponsor | Collaborator |
---|---|
Baylor College of Medicine | Southern Star Research Pty Ltd. |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Maximum VO2 | Maximum VO2 in ml/kg/minute will be collected via Exercise Stress Test. Range 30-85, higher is better. | 4 Months | |
Secondary | Mean systolic blood pressure | mmHg, range 20-200, both low and high are abnormal, goal is age, sex and height-based, goal 10-50 percentile | 4 Months | |
Secondary | Mean diastolic blood pressure | mmHg, range 20-150, both low and high are abnormal, goal is age, sex and height-based, goal 10-50 percentile | 4 Months | |
Secondary | Mean pulse pressure | mmHg, systolic blood pressure minus diastolic blood pressure, range 30-70 mmHg, goal is normal range for age and sex | 4 Months | |
Secondary | Weight | kg, range 50-300kg, lower is better, excluding underweight patients | 4 Months | |
Secondary | BMI | kg/m2, lower is better generally, excluding pts with BMI <5% for age | 4 Months | |
Secondary | Left ventricular strain by cardiac MRI | Continuous measure derived from post-processing MRI | 4 Months | |
Secondary | Right ventricular strain by cardiac MRI | Continuous measure derived from post-processing MRI | 4 Months | |
Secondary | Aortic root strain | Continuous measure %, higher is less stiff, Range 0-40 | 4 Months | |
Secondary | Aortic Root Distensibility | ×10-3 mm Hg-1, Continuous measure, range 0.1-10 | 4 Months | |
Secondary | Aortic Root ß-Stiffness index | No units, Range 0.1-90 | 4 Months | |
Secondary | Maximum aortic root dimension | Measured in cm, range 1-8cm | 4 Months | |
Secondary | Aortic root z-score | No units, based on body surface area published references, range -3 to 25 | 4 Months | |
Secondary | Aortic pulse wave velocity from MRI | meters/second, range 0-30 | 4 Months | |
Secondary | Pulse wave velocity derived from applanation tonometry | meters/second, range 0-30 | 4 Months | |
Secondary | Quality of Life Scale (QOLS) (ages 19-21 y) | 16 items, each with 7 point Likert, higher is worse | 4 Months | |
Secondary | Pediatric Quality of Life Scale (PedsQL) scale scores | reported in 3 domains, each reported on Likert scale, scaled to 0-100 scale, lower is worse | 4 Months | |
Secondary | Patient Health Questionnaire-9 (PHQ-9) | Nine-item questionnaire that detects depression and the severity of the depression, scaled from 0-27, higher is worse | 4 Months | |
Secondary | Patient Health Questionnaire-4 (PHQ-4) Subset A: Anxiety | Four-question test for anxiety and depression, scaled from 0-12 Subset A (Anxiety): Questions 1 & 2, which have a total PHQ 4 score range of 0 to 6. A score of 3 or greater is considered positive for screening purposes. | 4 Months | |
Secondary | Patient Health Questionnaire-4 (PHQ-4) Subset B: Depression | Four-question test for anxiety and depression, scaled from 0-12, Subset B (Depression): Questions 3 & 4, which have a total PHQ 4 score range of 0 to 6. A score of 3 or greater is considered positive for screening purposes. | 4 Months | |
Secondary | Generalized Anxiety Disorder 7-item (GAD-7) | Seven-item questionnaire that detects generalized anxiety disorder and the severity of the anxiety. GAD-7 total score for the seven items ranges from 0 to 21. Using a cut-off of 8 the GAD-7 has a sensitivity of 92% and specificity of 76% for diagnosis generalized anxiety disorder. | Every 2 Weeks for 4 Months | |
Secondary | Pediatric Symptom Checklist Parent (PSC) | The Pediatric Symptom Checklist (PSC) is a 35 item questionnaire that helps identify and assess changes in emotional and behavioral problems in children. A positive score on the PSC or Y-PSC suggests the need for further evaluation by a qualified health. PSC-35-Youth, English & Spanish cut off score is, 30 or higher. In a Mexican-American sample, a cutoff score of 12 was shown to be most sensitive. | 4 Months | |
Secondary | Pediatric Symptom Checklist Child (PSC-Y) | The Pediatric Symptom Checklist (PSC) is a 35 item questionnaire that helps identify and assess changes in emotional and behavioral problems in children. A positive score on the PSC or Y-PSC suggests the need for further evaluation by a qualified health. PSC-35-Youth, English & Spanish cut off score is, 30 or higher. In a Mexican-American sample, a cutoff score of 12 was shown to be most sensitive.
PSC Subscales: Attention Problems, Internalizing Problems, Externalizing Problems |
4 Months | |
Secondary | Pediatric Symptom Checklist Child (PSC-Y): Attention Subscale | The Pediatric Symptom Checklist (PSC) is a 35 item questionnaire that helps identify and assess changes in emotional and behavioral problems in children. A positive score on the PSC or Y-PSC suggests the need for further evaluation by a qualified health. PSC-35-Youth, English & Spanish cut off score is, 30 or higher. In a Mexican-American sample, a cutoff score of 12 was shown to be most sensitive.
PSC Subscales: Attention Problems Subscale: Children with subscores greater than or equal to 7 usually have significant impairments in attention. |
4 Months | |
Secondary | Pediatric Symptom Checklist Child (PSC-Y): Internalizing Problems Subscale | The Pediatric Symptom Checklist (PSC) is a 35 item questionnaire that helps identify and assess changes in emotional and behavioral problems in children. A positive score on the PSC or Y-PSC suggests the need for further evaluation by a qualified health. PSC-35-Youth, English & Spanish cut off score is, 30 or higher. In a Mexican-American sample, a cutoff score of 12 was shown to be most sensitive.
PSC Subscales: Internalizing Problems Subscale: Children with subscores greater than or equal to 5 usually have significant impairments with anxiety or depression. |
4 Months | |
Secondary | Pediatric Symptom Checklist Child (PSC-Y): Externalizing Problems Subscale | The Pediatric Symptom Checklist (PSC) is a 35 item questionnaire that helps identify and assess changes in emotional and behavioral problems in children. A positive score on the PSC or Y-PSC suggests the need for further evaluation by a qualified health. PSC-35-Youth, English & Spanish cut off score is, 30 or higher. In a Mexican-American sample, a cutoff score of 12 was shown to be most sensitive.
PSC Subscales: Externalizing Problems Subscale: Children with subscores greater than or equal to 7 usually have significant problems with conduct. |
4 Months | |
Secondary | PROMIS Pediatric Self- Reported Psychological Stress | PROMIS scores have a mean of 50 and standard deviation (SD) of 10 in a referent population. On the T-score metric, A score of 40 is one SD lower than the mean of the reference population. A score of 60 is one SD higher than the mean of the reference population. Better health is a lower score and worse health scores higher. | 4 Months | |
Secondary | PROMIS Parent Proxy Reported Psychological Stress- Parent Proxy | PROMIS scores have a mean of 50 and standard deviation (SD) of 10 in a referent population. On the T-score metric, A score of 40 is one SD lower than the mean of the reference population. A score of 60 is one SD higher than the mean of the reference population. Higher score is worse health. | 4 Months |
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